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Stopping Migraines Before They Hurt: NeurAxon Pursues New Pain Drug

Nobody has yet come up with a drug that can stop the pain from migraine headaches before it starts. By this time next year, the people at Waltham, MA-based NeurAxon will know if they have created the first drug that works for the roughly one-third of patients who get a serious inkling before migraines kick in that pain’s on the way.

Migraine headaches affect about 30 million people in the U.S., so any powerful new drug aimed at helping this group has a big opportunity. NeurAxon’s lead pill in development is designed to combine the pain relief of a standard drug from the family of “triptans,” along with the more novel ability to inhibit another enzyme called nNOS that controls pain sensations. Blocking this particular enzyme is important because no other pain drug specifically hits it, and the target is associated with what is known as “aura.” These are the flashes of light in people’s vision, the zigzagging patterns, or blind spots that about one-third of migraine sufferers get about 30 to 90 minutes before pain sets in. Early clinical trials suggest that NeurAxon’s drug might be the first that patients could take when the “aura” comes on, which means the drug could start working in time to fight the pain before it starts.

“If we can prove that we can dose at aura, it’s a fundamental breakthrough,” Bloch says. “There’s never been a drug like that.”

The current state-of-the-art “triptan” family of seven drugs generates about $3 billion a year in worldwide sales, Bloch says. The leader of the class is GlaxoSmithKline’s sumatriptan (Imitrex), which is scheduled to face competition from cheaper generic copies next year. (Although Glaxo may have protected its market a while longer with a new combo of sumatriptan with the pain reliever naproxen, called Treximet, that won FDA approval in August). Drugs in this class are not a panacea, because they have to be taken once a patient already feels migraine pain, and then they offer some relief for 50-75 percent of patients within two hours. They don’t last a full 24 hours, allowing room for so-called “rebound” headaches. The drugs also come with a warning that they can cause high blood pressure and other heart side effects like stroke.

NeurAxon, which got started in 2004, has built its secret sauce around compounds designed to last longer in the body, and be more effective by specifically hitting that extra neuronal nitric oxide synthase (nNOS) enzyme. Scientists have long known the enzyme is intimately related to pain sensations, but the target has stymied drug developers because it is so structurally similar to other families of enzymes. If you hit too many of the related enzymes, it can lead to damaging heart effects, Bloch says.

The company has completed a clinical trial in 192 patients has shown that NeurAxon isn’t causing dangerous heart affects at any dose tested, Bloch says. Now it’s time for the tougher exam. Does it work any better, is it really safe for the heart in repeated studies, and does the pain relief last long enough?

This is a pivotal year for answering those questions. NeurAxon has lined up three mid-stage clinical trials. The first … Next Page »